The National Drugs for Rare Diseases (DRD) Strategy Needs a Reframe.
ImmUnity Canada and the Network for Rare Blood Disorder Organizations (NRBDO) have collaborated to create a report that amplifies the rare blood disorder patient perspective and proposes a reframe for Canada’s DRD Strategy.
The Rare Blood Disorder Community Perspective
While Canada is making progress in addressing issues facing the rare disease community, currently, the interests and needs of patients with rare blood disorders are not well represented in the rare disease discourse.
The rare blood disorder community and rare disease patients who rely on therapies derived from blood and plasma or their substitutes are in a unique situation—including very limited benefit from a common DRD formulary—and merit recognition as a distinct voice within the rare disease patient community.
Reframing the National Drugs for Rare Diseases Strategy
The National DRD Strategy has been created to address the unmet needs in the rare disease community and the high cost of rare disease drugs to provincial and territorial drug plans.
With the framing as a problem of expensive drugs for rare diseases, the solution that has emerged is that, of the $1.5B pledged for the strategy over 3 years, $1.4B will be used to create and fund a common list of drugs across the provinces and territories through bilateral agreements. This introduces many challenges, and as the timeline is currently unknown, the funding of $1.4B is unused and not benefiting patients.
Reframing the objective as moving us toward timely and equitable access to treatment and care for rare disease patients allows the patient community, clinicians, researchers, and policymakers alike to reimagine a role for the federal government to:
- support research and data collection, including clinical trial sites and patient registries
- create a National Newborn Screening Program
- engage with manufacturers to bring novel diagnostic and therapeutic technologies to Canada
- improve timelines and lessen uncertainty in our health technology assessment processes
- provide clear guidance to the provinces about reimbursement for new indications for approved therapies, including off-label usage
We believe that the optimal role of the federal government is to create a national infrastructure that supports timely and equitable access to treatment and care for rare disease patients.
Recommendations
- Include representatives from the rare blood disorder community more inclusively in the DRD process, including on the Implementation Advisory Group (IAG). Leverage patient partners to co-develop plain language summaries and education resources and tools.
- Expand the scope of CDA-AMC’s recommendations to provide clear guidance to the provinces on reimbursement for new indications on approved therapies, including off-label usage, where generics and biosimilars are being introduced, and therapies that have been proven effective but that manufacturers have not deemed profitable enough to bring to the Canadian market.
- Consider the capacity and resources of small rare disease patient organizations when designing processes for their input, from HTA submissions to RFPs, and where possible, provide assistance, including infrastructure to support participation.
- Review disease states where IVIG is currently being used as a first-line treatment and where more effective and potentially more cost-efficient treatment options are available.
- Expand the use of therapies already available in Canada to include off-label or second-line indications, allowing for more equitable and timely access for rare disease patients.
- Encourage provinces and territories to follow CDA-AMC’s reimbursement recommendations without imposing additional restrictions when considering reimbursement for therapies, improving equity across jurisdictions.
- Establish an expert advisory committee to explore the feasibility of a Canadian infrastructure of publicly-funded point-of-care gene therapy technology to make these therapies more accessible to more Canadians.